Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease

scientific article published on 17 September 2014

Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1016/J.YMGMR.2014.08.004
P932PMC publication ID5121362
P698PubMed publication ID27896112
P5875ResearchGate publication ID265731621

P50authorYoichi IshitsukaQ73209250
Muneaki MatsuoQ87702543
P2093author name stringTetsumi Irie
Hidetoshi Arima
Hirohito Doi
Junya Eto
Koki Shraishi
Koki Wada
Miyuki Maeda
Sakiko Mochinaga
Tatsuhiro Mizoguchi
P2860cites workChronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progressionQ21142752
Niemann-Pick disease type CQ21202883
2-hydroxypropyl-beta-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C diseaseQ24598867
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 diseaseQ27006029
Development and validation of sensitive LC-MS/MS assays for quantification of HP-β-CD in human plasma and CSF.Q33825055
Therapeutic potential of cyclodextrins in the treatment of Niemann-Pick type C diseaseQ34069183
Pharmaceutical applications of cyclodextrins. III. Toxicological issues and safety evaluationQ34417057
Gamma-secretase-dependent amyloid-beta is increased in Niemann-Pick type C: a cross-sectional studyQ34555434
Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouseQ34930699
Normalization of cholesterol homeostasis by 2-hydroxypropyl-β-cyclodextrin in neurons and glia from Niemann-Pick C1 (NPC1)-deficient miceQ35841853
Miglustat treatment may reduce cerebrospinal fluid levels of the axonal degeneration marker tau in niemann-pick type C.Q36433310
Effects of cyclodextrin in two patients with Niemann-Pick Type C diseaseQ40265254
Genetic and chemical correction of cholesterol accumulation and impaired autophagy in hepatic and neural cells derived from Niemann-Pick Type C patient-specific iPS cells.Q40473012
Unesterified cholesterol accumulation in late endosomes/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartmentQ42140225
The pharmacokinetics of beta-cyclodextrin and hydroxypropyl-beta-cyclodextrin in the ratQ70291442
P407language of work or nameEnglishQ1860
P304page(s)391-400
P577publication date2014-09-17
P1433published inMolecular Genetics and Metabolism ReportsQ27725363
P1476titleEffects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease
P478volume1